FDA Grants Orphan Drug Status to Acceleron's PAH Candidate

Acceleron Pharma Inc.XLRN announced that the FDA has granted an orphan drug designation to its investigational candidate sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH), a rare disease of highly unmet medical need.

Notably, the orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. This status also makes the company entitled to certain other benefits including tax credits and grant funding related to clinical study expenses. The tag also makes sotatercept eligible for seven years of marketing exclusivity in the United States upon potential approval from the regulatory body.

Sotatercept is currently being evaluated in two phase II studies for treating PAH. In June, Acceleron completed enrolling patients in the PULSAR study and plans to report top-line data from the same in the first quarter of 2020. Also, the company is currently recruiting subjects in the SPECTRA exploratory study to have a better understanding of sotatercept's potential impact on PAH. Preliminary results from this study are expected in 2020.

Shares of Acceleron were up almost 2.2% following this news on Monday. In fact, so far this year, the stock has gained 4.5% against the industry’s decline of 2.5%.

We would like to remind investors that Acceleron along with global collaboration partner and biotech giant Celgene Corp. CELG is developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia and myelofibrosis.

In June this year, Celgene announced that the FDA accepted its biologics license application (BLA) for luspatercept to treat adult patients with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia, who have ring sideroblasts and require red blood cell (RBC) transfusions, and those with beta-thalassemia-associated anemia requiring RBC transfusions.

The FDA has now granted a Priority Review to the BLA for the treatment of beta-thalassemia and also set an action date of Dec 4, 2019. In addition, the regulatory agency has set a target date of Apr 4, 2020 for the evaluation of the MDS indication.

Notably, Acceleron received a $25-million milestone payment from Celgene post the FDA’s acceptance of the BLA for luspatercept. The company is entitled for further milestone fees if the regulatory body approves luspatercept for any of the above-mentioned indications.

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